LAUSR

Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance

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1 Published in 2019 at "Neurology and Therapy"

DOI: 10.1007/s40120-019-00157-4

Abstract: Alglucosidase alfa received marketing approval for the treatment of Pompe disease in Japan in 2007. We conducted a post-marketing surveillance study to monitor the long-term safety and efficacy of alglucosidase alfa therapy among Japanese patients… read more here.

Keywords: safety; japanese patients; marketing; pompe disease ... See more keywords

Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for a patient presenting with a PRKAG2 mutation.

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0 Published in 2017 at "Molecular genetics and metabolism"

DOI: 10.1016/j.ymgme.2016.09.006

Abstract: OBJECTIVE PRKAG2 syndrome, an autosomal dominant disorder, is characterized by severe infantile hypertrophic cardiomyopathy and heart rhythm disturbances to cases with a later presentation and a spectrum of manifestations including cardiac manifestations, myopathy and seizures.… read more here.

Keywords: alfa enzyme; pompe disease; alglucosidase alfa; mutation ... See more keywords

A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease

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1 Published in 2022 at "Frontiers in Pharmacology"

DOI: 10.3389/fphar.2022.903488

Abstract: Background: A high prevalence of infantile-onset Pompe disease (IOPD) in the Chinese population has been noted, but there are currently no reported clinical trials of enzyme replacement therapy (ERT) for IOPD in this population. The… read more here.

Keywords: efficacy safety; alglucosidase alfa; study;